Beyond Achondroplasia

Growing together with Clara

October 20, 2017
by inesp.alves
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Recruitment in clinical trials

The following document, shared by BioMarin in September 2017, is a clarification on the ongoing clinical trials for achondroplasia and was released for associations and families: BioMarin Achondroplasia Programme Update 20Sept17 It is important to mention that families interested in … Continue reading

June 3, 2017
by inesp.alves
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BioMarin updates the Multicenter and Multinational Clinical Study 111-901

The “Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia” is a prospective observational study. This study’s code name is 111-901. The aim of this study is to collect systematic growth measurements of the children in order to collect data … Continue reading

May 12, 2017
by inesp.alves
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Phase 3 trial for achondroplasia – BioMarin Study 111-301

The following information is fully available at the European Union Clinical Trials Register. Some sections will be here highlighted: 1. Full title of the trial A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN … Continue reading

November 23, 2016
by inesp.alves
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A challenging report on Vosoritide

A rare disease encompasses any life-threatening or chronically debilitating disorder or condition which, as the name suggests, is uncommon in the general population. Rare diseases typically exhibit a high level of symptom complexity and as a result they very frequently require … Continue reading

November 7, 2016
by inesp.alves
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Conference on achondroplasia

In a collaboration initiative between ALPE Foundation, FIMABIS and Dr. Felipe Luna, Head of Orthopedic Surgery and Traumatology at  Málaga Central Hospital, Spain, the Second Conference of Malaga Achondroplasia Unit “Around achondroplasia – research for families” will be held in … Continue reading

October 20, 2016
by inesp.alves
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BioMarin presents report on phase 2 study of Vosoritide

BioMarin presented on the 19th October 2016 a press release on the data for Vosoritide in phase 2,  during the ASHG 2016 meeting. There is no overall novelty in this latest press release (19th Oct 2016) when comparing to the previous press … Continue reading

May 17, 2016
by inesp.alves
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Questions and answers – BMN-111 Drug Trial

LP Doc Talk is a project of Growing Stronger, a foundation based in S. Francisco, California and founded by Amer and Munira Haider, parents of Ahmin,a boy now with 7 years-old, born with achondroplasia. When I started to search about achondroplasia, I … Continue reading

May 13, 2016
by inesp.alves
3 Comments

BMN-111 Phase 2: study 111-202 and extension study 111-205

Study 111-202 A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children with Achondroplasia (ACH) This study is enrolling participants by invitation only   Estimated Enrollment: 46 Study start date: January 2014 Estimated Study Completion … Continue reading

April 14, 2016
by inesp.alves
1 Comment

LP Doc talk – About BMN-111

  Growing Stronger provides LP Doc talk, a series of online, live conversations with world-leading doctors and other experts focused on dwarfism. You can join live online for each session or visit the archived recordings anytime thereafter. This is a extremely … Continue reading

March 7, 2016
by inesp.alves
2 Comments

Roadmap for a new therapy – by BioMarin

BioMarin created a very clear and easy to read infographic poster about BMN-111, now called vosoritide. Here is just the first part, but it is worth seeing the complete infographic. It also explains the 4 different phases of a clinical trial. … Continue reading

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